Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, CF, mRNA
Brief summary
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Detailed description
This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1), the safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF (Part 2), and the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF (Part 3).
Interventions
DRUGRCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
OTHERPlacebo
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
DRUGIvacaftor
ivacaftor administered orally for 6 weeks
Sponsors
ReCode Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
Participant and investigator masking only applies to Part 1 which is randomized. For Part 2 and Part 3, there is no masking, and this part is Open Label.
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
Yes
Inclusion criteria
Part 1 Major Inclusion Criteria: * Healthy, adult, male or female, 18-55 years of age, inclusive, at screening. * Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive * The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted * The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination findings at screening. * Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol. Part 1 Major
Exclusion criteria
* History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease. * The participant has supine blood pressure (BP) \>150 mm Hg (systolic) or \>90 mm Hg (diastolic), following at least 5 minutes of supine rest. * The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory. * The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with greater than 10 pack years of smoking history are excluded. Part 2 Major Inclusion Criteria: * Confirmed diagnosis of CF * Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height * a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR * b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications Part 2 Major
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). | From Baseline Through Day 29 | Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) |
| Part 2: The number of participants with CF with AEs and SAEs. | From Day 1 through Safety Follow-up, Week 24 | Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF |
| Part 3: The number of participants with CF with AEs and SAEs. | From Day 1 through Safety Follow-up, Week 24 | To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF. |
Countries
France, Netherlands, New Zealand, United Kingdom, United States
Contacts
STUDY_CHAIRJohn Matthews, MBBS, MCRP, PhD
ReCode Therapeutics, Inc.
Outcome results
None listed